SUPPLY CHAIN ARTICLES
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Defining The RNA Therapeutics Industry In 2023
Four executives outline which industry challenges, strategic considerations, and opportunities should be top of mind as RNA therapeutics companies establish their to-do lists for 2023.
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ARW's ATMP Manufacturing Must-Reads (ST. PATRICK'S DAY EDITION!)
Day-in and day-out, I write, read, listen to, and watch as much content as I can about CGT and RNA therapy manufacturing, in particular, and/or other ATMP industry-related topics that you should at least be aware of in the manufacturing facility. Once a month, I compile the articles and industry updates I think are most worthy of your time into an unconventional roundup article.
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A "Hot Take" on Personalized mRNA Medicines
mRNA’s small-scale and cell-free production are often-touted benefits in the personalized medicines sphere. But as I reviewed my notes from the BioPhorum ATMP member event, I found myself coming face to face with a difficult truth.
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Inside Alnylam's Playbook For High Volume siRNA Production
As one of the first companies to establish a chemoenzymatic ligation platform, Alnylam's Maines and Nechev are in the perfect position to espouse wisdom on the practical aspects and remaining challenges of implementing enzymatic ligation at a large scale. Here in this installment, Maines and Nechev outline what they’ve learned thus far about the science of enzymatic ligation, while also paying credence to the unknowns and existing barriers that, as a frontrunner, Alnylam will inevitably (but willingly) be tasked with facing in the future.
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Therapeutic Oligonucleotides: Regulations & Quality Standards
Oligonucleotides have surfaced in the past few years as promising therapeutic agents to treat diseases such as neurodegenerative disorders, respiratory disorders, cancer, and diabetic retinopathy. This article recaps a recent USP workshop where participants from regulatory agencies, industry, and academia gathered to discuss best practices and perspectives.
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Picasso, Pick-Up Lines, & Personalized Medicine: mRNA's Path Forward
Overall, my presentation on what the mRNA industry can learn from the artist Picasso singled out a few specific areas from which I felt our industry could garner greater inspiration and operational prowess. But given how “collaborative” many of these sources of inspiration are, I’ve introduced each of my takeaways with a surprisingly relevant (albeit bad) art-themed pick-up line. After all, if there’s one sector that knows it takes more than one to “tango,” it’s the mRNA industry.
ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
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Ensuring consistent replication and proper retention of plasmid DNA sequence is vital to the economics and efficiency in generating starting materials used for production of AAV delivered gene therapy and mRNA vaccines and therapeutics.
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Learn how a robust understanding of the RNP’s critical process parameters and CQAs is crucial for ensuring product consistency, enhancing patient safety, and maintaining regulatory compliance.
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Single-use technology is vital for ensuring the quality and expedited market entry of genomic medicines. Learn how LNP technology is paving the way for a new era of personalized, precise medicine.
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As gene therapy organizations are asked to balance the need for rapid clinical progression and stringent quality expectations, explore how to optimize plasmid production and reach patients with efficiency.
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We report on the use of a novel lipid nanoparticle (LNP) reagent in a validated protocol to achieve successful complex gene editing in primary T cells with high efficiency while maintaining high cell viability.
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This review suggests technical remedial options for the production of pDNA and focuses on mRNA synthesis, purification, and encapsulation.
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Explore a program focused on achieving process consistency through risk mitigation in four key areas: raw materials sourcing, equipment, manufacturing processes, and finished product testing.